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Current Gene Therapy

Editor-in-Chief

ISSN (Print): 1566-5232
ISSN (Online): 1875-5631

Applications of Muscle Electroporation Gene Therapy

Author(s): Shulin Li and Michael Benninger

Volume 2, Issue 1, 2002

Page: [101 - 105] Pages: 5

DOI: 10.2174/1566523023348183

Price: $65

Abstract

Muscle is a convenient and accessible site for non-viral gene delivery, which can manufacture gene products and provide a long-duration of gene expression. The level of gene expression after administration of naked DNA plasmid or polymer-formulated DNA plasmid containing a reporter gene to muscle via syringe injection, however, is very low. As a result, no significant therapeutic effect can be detected after saline- or polymer-mediated gene delivery into muscle. In 1998, investigators published a striking new approach-electrotransfection-for intramuscular gene delivery (now commonly referred to as electroporation or electroinjection). Electroporation of a non-viral gene into the muscles of small animals has increased the level of gene expression by as much as two orders of magnitude, which is comparable to levels achieved with adenoviral gene delivery. Three years later, intramuscular electroporation gene delivery technology has blossomed. Treatments for different diseases using this approach in animal models have been reported. In this review, I discuss the applications of intramuscular electroporation gene therapy to treat malignancies, renal disease, and anemia, and to prevent drug toxicity to sensory nerves.

Keywords: Muscle Electroporation, Gene Therapy, polymer-formulated DNA, Erythropoietin (EPO), Chimera Gene, Cis-diaminedichloroplatinum


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